Chengdu Hongji Biotechnologies Co., Ltd.

VAN-2201 is Phase I clinical trial to assess the safety and tolerability of KH631 in subjects with neovascular AMD. KH631 is gene therapy designed to deliver a protein which targets and blocks VEGF via an adeno-associated viral vector. The standard of care for patients with neovascular AMD are anti-VEGF treaments, which have demonstrated improvement in vision and reduction in fluid. A one time placement of a product which inhibits VEGF has the potential to reduce the patient burden of regular intraocular injections.

1期主要评估视网膜下注射不同剂量KH631眼用注射液在新生血管性（湿性）年龄相关性黄斑变性（nAMD）患者中的耐受性，初步评估KH631眼用注射液治疗nAMD的疗效，主要在24周评估总体安全性。2期主要根据1期耐受性、安全性和初步疗效评估的结果，选择2期推荐剂量组扩大样本量评估KH631眼用注射液在nAMD患者中的疗效和安全性，主要在52周评估有效性。长期随访期目的为在已接受KH631眼用注射液视网膜下注射的患者中评估KH631眼用注射液的长期安全性和有效性。

KH631 is a adeno-associated virus (AAV) vector-based gene therapy for subretinal injection. The long-term, stable therapeutic protein after one time injection for nAMD could potentially reduce the treatment burden and maintain vision.